Sankaran and Orkin showed that BCL11A suppresses production of fetal hemoglobin. Their landmark publication in Science kicked off a new era for sickle cell disease research. Just three years later ...

A new review in Engineering reveals that CRISPR technologies are revolutionizing regenerative medicine. Scientists from ...

Learn how CASGEVY, the first NHS-approved CRISPR gene therapy, treats sickle cell disease through a revolutionary genetic ...

Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient's bone marrow to start producing red blood cells with ...

in red blood cells, by introducing a gene known as BCL11A which down-regulates the production of the adult form of the oxygen-carrying molecule. Reverting to HbF in thalassaemia and SCD patients ...

A recent review published in the journal Engineering delves into the significant advancements and potential of CRISPR technologies in the field of ...

There, the CD34+ cells are isolated. Then, the CRISPR associated protein 9 is used to edit the BCL11A gene before the cells are frozen. The edited cells are returned to the body in a single infusion.